IAmA parent of a 3-year-old with cystic fibrosis joined by two pediatric lung doctors. We are concerned about the high cost of new cystic fibrosis medicines. Ask us anything!

Thank you Doctors O'Sullivan and Orenstein for all you do! I'm 27 with CF, still kickin'.

Kalydeco and Orkambi are great additions to treatments, but our existing treatments and meds are still so expensive, what can we do to get those costs to go down?

Hey Rosie, thanks for signing on and kickin' it with a q! I'm curious (if it's not too personal of a question) of what the financial impact is for CF meds and care later on. My son is 3. We have been pretty guarded by insurance thus far, but with three hospitalizations in three years it's cost about $9,500 not including doctor visits and some co-pays. I'm going to do anything to keep Eli as well as possible and I want to pay a fair price for a good service, but anyhow, I'm just curious about what it costs for an older person.

That's the $64000 (or more like $300,000) question. We're hoping that public opinion, and maybe even some federal regulation might help convince Vertex and other companies that they can continue to develop these wonderful new drugs, and make a nice profit without bankrupting families and the whole system.

If one looks at the mission statements of the founders of these companies their emphasis was on doing good and making a fair profit from doing good work. Now (it seems to me) the emphasis is on shareholder profits and CEO compensation, not on the patient's well being. The philosophy seems to have flipped to "Let's make a big profit and if we do some good along the way, fine."

I sense this as well.

We know that out of the thousands of potential molecules, only a handful make it to clinical trials. Out of that handful, only a few obtain FDA approval. Out of those, maybe one drug will pay it's own R&D costs through sales. It's essentially costing billions to bring a single drug to market, so manufacturers have to set high costs to recoup their investment.

It is also not sensible profit-wise to research and manufacture a treatment for a more rare condition. Fewer sales would not be enough to recoup those costs.

Now, other developed countries can get the same drug cheaper because of governmental negotiaton contracts and less economic power (whatever their market will bear), and this is possible because the U.S. pays higher prices for drugs. So in essence, the U.S. is subsidizing costs for the rest of the world.

We also know that drug companies spend tons of money to advertise their products directly to consumers; as I've heard someone else put it, "In the rest of the world, doctors tell their patients what to take, but in America, patients tell their doctors what to prescribe." I am of the opinion that direct advertising to consumers should not happen with pharmaceuticals.

What are your thoughts on this?

Hello! R&D is most certainly incredibly expensive and cystic fibrosis rare and not a money maker. That's why the cystic fibrosis community has for years raised millions in charity dollars to entice companies like Vertex to commit to the research. Vertex has also used lots and lots of its own money to bring two medicines to market. That being said, the drugs that have come out of the work cost more than a home for one year's worth of medicine. Orkambi costs around $259K/year and Kalydeco somethng like $376K/year from a specialty pharmacy. Drug companies that address rare disease are given a monopoly on the license for a period of time - I need to double check but it's something like seven years. Given the taking of charity dollars and the monopoly, there needs to be a lot more transparency before I can accept that meds have to be priced the way they do. What's the profit margin on a single pill? Has the company made back its billions? How much of the profits go to shareholders versus back into R&D? I've asked Vertex and I'm still waiting for a response to these questions. I'm willing to pay more and subsidize to an extent - but when a drug like Orkambi improves lung function by 3 percent and costs more than a house, what's the company going to charge for a cure? A million? Life is priceless but companies should be prevented from extorting people. The other thing is, people with cystic fibrosis abroad were given these medicines in trial and now can't have them because they are full-tilt too expensive, even at a reduced rate. That's cruel and out of line with the way the drugs were funded.

There is a fair amount of debate about just how much it costs to get a drug to market. Some estimates are a billion dollars or more, others are much, much lower. Unfortunately, there is very little transparency in drug pricing. Many companies roll in non-research costs (such as the advertising you allude to) in the so-called "cost" of R&D. Without more information it is very hard to know what a fair price for a medication is.

Exactly. More transparency is needed. Drug companies with a monopoly on new meds should be held to account.

How do you balance the hope and realism?

This is tough. As a physician I try to be realistic, but given the advances that have been made over the last 20 years and the hopes that we have for even better therapies soon, it is much easier to be hopeful now than it was in the 1980s. But that hope is coming at a huge monetary cost and this is the issue. The UK won't even approve Orkambi for their CF patients because of the cost,

Ireland, Scotland and England are all no-gos for the latest.

Oooh that is such a goooood question. As a parent, I have to have hope that my son will live a good life and my hope of all hopes is that his disease will be cured. (Without bankrupting or financially straining the heck out of my family/him...that's also part of that hope). Realistically, he could be hospitalized quite a lot. He's had two gut surgeries and a five-day hospitalization to treat a lung infection so far, plus a good number of 21-day antibiotic rounds in three years. It's a progressive illness, so he's well now despite what I just described. He's a happy little fellow. The reality of his disease hits me when it flares up. Any time something new crops up it feels like a wake-up call and a reminder of what my son is dealing with. We just try to be happy and live day-to-day and carry on like life is normal - because to us, it is!

Being a CF patient or parent of a child with CF is tough work. There are multiple medications to take every day. Chest physiotherapy for at least 20 minutes twice a day. Multiple inhaled medications every day. Pills with everything you eat in order to be able to digest the food. What we physicians ask parents/patients to do to stay healthy is daunting. That you all do it is amazing. The hope is that all this work turns into years of "normal" life. But again - to stay on topic - this is all incredibly expensive.

Indeed! While we are grateful for Eli to have the level of care he has had, the surgeries and hospitalizations have cost thousands of dollars out of pocket. (Which I'm proud to say are paid up). Drugs costs are one tentacle of the greedy 100-armed octopus that is American healthcare.

What misconceptions do you find people often have about Cystic Fibrosis?

Major misconception = that CF is a fatal disease for children. In 2016 more than half of people with CF in the USA are over 18 years of age.

I am thankful for that stat!

Hi! People don't generally know what it is. I know I didn't when I got the diagnosis for my son at two weeks. The shortest definition is that it is a fatal genetic lung illness. Of course it's much more complex than that. I've heard all kinds of untrue things about the disease. Often older people with cystic fibrosis will encounter ignorance. It seems to come in the form of judgement and scowls at a coughing fit, for instance. It's not contagious and the bacteria that mess up the lungs won't get a person with a 'normal' genome sick. I could go on...what haven't I covered?

First, just want to say I have a younger brother with cf who is now on Orkambi and doing extremely well. I'll be praying for your 3-year-old and doing what I can to make cf stand for cure found.

Is there a specific treatment being developed that you are really excited about?

Hello! I'm excited about Orkambi! My son has two mutations of the DeltaF508, so that is one that could help him. That being said, since the drug is so new and he's well now, I doubt I would have him join a trial for younger children unless something drastic changes with his health. I'm really excited as well about the various trials and three-drug combos. I'm not so excited about the costs of these medicines. Journalists are the worst boosters for causes. We always see problems. Not everyone is having a positive experience with Orkambi. However, as a mom I'm thrilled that progress is being made.

Hi there. Thanks for the AMA. My newly adopted son tested psitive for the famous 508 mutation but not the other major ones screened. Two sweat tests (one on each leg at same time) showed very low numbers, not even in the "gray zone" so I know he is a carrier and doesn't have CF. I've been told by the geneticist not to worry about it aside from family planning later on.

My question is this...is that really it? Was my two week nightmare waiting for the sweat tests over? Is there any research showing the one mutation causes anything else that is serious?

Hi! Thanks for weighing in. If he has only one copy of the mutation he should be just fine! I have one copy and my husband has one copy. Because we each have a copy, there is a 1/4 chance when we have a child that that child will have CF. We also have a daughter who doesn't have CF, but like your son, we will have her tested to see if she is a carrier just so she knows for later on.

Why are you here and not in a european country with national healthcare?

I was born here.

Adding: We are debating the cost of the medicine not who pays tonight. The script needs to be flipped.

If money is an issue go where its free.

Hi! I'd rather stay in my home country and improve the system than move. Also, the high cost of medicines is a tax on society. For instance, when a cystic fibrosis drug costs $259K/year, which is the case for a new drug called Orkambi, taxpayers will pay for that. Consumers with private insurance pay too since the buck is always passed to the little guy. The skyrocketing costs of our medicines is happening with antibiotics, cancer and genetic disease medicines, etc. Leaving doesn't address the problems at home.

Shouldnt you be more worried about your own kid?

If we all only worry about ourselves...the world kind of sucks.

Perhaps we should be asking why we don't have national healthcare here in our own wealthy country.

In Canada people with cystic fibrosis live longer than in the U.S. It does make one wonder!